United Therapeutics (UTHR) investor relations material

Key clinical and commercial milestones

• Reported best-ever clinical trial results for pulmonary hypertension and pulmonary fibrosis, with Tyvaso improving forced vital capacity more than any prior FDA- or EMA-approved medicine.

• Ralinepag demonstrated superior outcomes in slowing clinical worsening and improving patient status in pulmonary hypertension, with expectations for rapid adoption post-approval.

• Anticipates FDA filing for ralinepag and Tyvaso in IPF by mid-2024, with approvals targeted for 2025 and major launches by 2027.

• Peak revenue for ralinepag projected at five times launch revenue, with initial $3 billion from 15,000 patients and further growth from broader patient populations.

• Tyvaso expected to reach 30,000 pulmonary fibrosis patients within three years of launch, with potential for 10x launch revenue as indications expand.

Product innovation and pipeline expansion

• Introduced Trezist, a soft mist inhaler reducing cough by 90% compared to dry powder inhalers, with FDA filing planned for 2026 and launch in 2027.

• Pipeline includes once-daily inhalers, PRN rescue inhalers, and a triple-combination oral pill for pulmonary hypertension, with launches planned through 2028.

• Plans to transition Tyvaso and Trezist into additional pulmonary indications, leveraging easier regulatory pathways and improved patient tolerability.

Organ manufacturing and xenotransplantation progress

• Two active xenokidney INDs with first-in-human trials underway; initial patients show normal kidney function and no rejection.

• Next cohort of four patients to be completed by summer 2024, with BLA filing targeted for late 2028 and potential approval in 2029–2030.

• UThymoKidney combines kidney and thymus transplantation to promote immune tolerance, with first patient transplant expected within three months.